Global Head of GSK Rare Diseases, GSK House, 980 Great West Road, Brentford, TW8 9GS, UK.
Nat Rev Drug Discov. 2011 Jun 24;10(7):475-6. doi: 10.1038/nrd3493.
Changes in regulatory policy and legislative incentives to promote the development of drugs for rare diseases - orphan drugs - have led to increases in the number of orphan drug designations, but the rate of such products reaching the market remains frustratingly flat. This article highlights areas in which novel approaches could facilitate regulatory approval and access to treatments for rare diseases.
监管政策的变化和立法激励措施促进了罕见病药物（孤儿药）的发展，孤儿药的指定数量有所增加，但这些产品进入市场的速度仍然令人沮丧地缓慢。本文重点介绍了一些新方法可以促进罕见病的监管审批和治疗途径。
